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Issue Background

Patient Focused Drug Development

The Patient-Focused Drug Development (PFDD) initiative is an FDA-led initiative to expand and accelerate the integration of the patient’s voice into therapeutic development and regulatory review. Initially launched as one of FDA’s commitments under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), PFDD has grown substantially in recent years thanks to provisions in the 21st Century Cures Act, PDUFA VI, and FDA-initiated endeavors. 

There are several components of PFDD, each pushing drug development to be more patient-focused and patient friendly. The initiative includes Patient Focused Drug Development (PFDD) meetings, which are meetings that aim to help FDA understand the burden of disease from the patient perspective and to gain an appreciation for the factors that are taken into account by patients when a treatment is chosen.  At PFDD meetings, individuals impacted by a specific disease convene to share their personal experiences of living with the disease, and to share insights on treatment impacts that would be most important or beneficial.  The experiences and insights shared at the PFDD meetings inform the FDA about important considerations including risk-benefit of treatment options, the severity of the condition, and the urgency of finding treatments, which can then be incorporated into the drug development process and assessment of future therapies.

There are currently two types of PFDD meetings: (1) FDA-led and (2) externally-led.  The FDA-led meetings were the first type of PFDD meeting established by the FDA to learn about twenty specific disorders (none of the diseases under MDA’s umbrella were included).  In December 2015, the FDA indicated that a second type of PFDD meeting would be available where stakeholders could hold their own PFDD meetings and invite the FDA to engage in the meeting through a formal process.  This second type of PFDD meeting is referred to as and an externally-led PFDD meeting—meaning that stakeholders outside of the FDA are leading the meeting.  MDA is grateful to the FDA for offering the option of hosting externally-led PFDD meetings, and MDA has collaborated in the past with key stakeholders to hold several externally led PFDD meetings, including meetings for Friedreich’s Ataxia (FA), spinal muscular atrophy (SMA), Charcot Marie Tooth (CMT), and Mitochondrial diseases. 

In addition to PFDD meetings, FDA has also expanded the PFDD initiative to include the generation, collection, and submission of empirically collected patient reported outcomes (PROs), patient preference information (PPI), and patient experience data (PED). Each of these offerings allow outside entities, including patients and patient organizations, to submit these data to FDA for use in assessing the benefits and risks of a new therapy, as well as the potential impacts a new therapy may have on a disease population. 

The PFDD initiative also incorporates opportunities for communities to submit draft guidances to FDA on how best to develop therapies within their specific disease area. Already two communities within MDA’s space, the Duchenne muscular dystrophy and amytrophic lateral sclerosis communities, have worked with FDA on disease-specific guidances of this nature. 

The PFDD initiative continues to evolve, and MDA will seek to represent and support the patient voice in each and every one of the exciting opportunities FDA’s PFDD initiative has to offer. 

Future Meetings: None at this time

Past Meetings

Pompe PFDD Meeting

On July 13th, 2020, the Muscular Dystrophy Association (MDA) was pleased to convene the very first Pompe disease Patient-Focused Drug Development (PFDD) meeting. This virtual meeting offered individuals with Pompe and their loved ones an opportunity to inform the U.S. Food and Drug Administration (FDA), drug developers, and other key stakeholders on the daily experiences of living with Pompe disease and how they viewied the benefits and risks of treatments for Pompe. MDA was pleased to partner with the Acid Maltase Deficiency Association (AMDA) and United Pompe Foundation in this endeavor to ensure that the viewpoints of the community were well represented. 


MDA was proud to partner with the FSHD Society and other FSHD stakeholders to convene the FSHD Voice of the Patient Forum initially scheduled for April 21, 2020, but was rescheduled for June 29th, 2020 on a virtual platform due to the coronavirus pandemic.  We encourage all members of the FSHD community to visit the meeting's webpage and check out the meeting's outcomes.

FA PFDD Meeting

MDA is proud to have collaborated with three other national organizations to host the Friedreich’s Ataxia (FA) meeting that was held on June 2, 2017 and attended by more than 25 FDA officials, more than 25 medical researchers and those from pharma, and 140 patients and family members.  During the meeting, invited panels of speakers were selected to represent the diversity of the disease and briefly spoke on the impact of FA on their day-to-day lives, and what they would like effective treatments to achieve.  The information captured at the meeting was published in a document called The Voice of the Patient Report and submitted to the FDA to help evaluate future FA therapies — including what will be the first therapy for FA.

SMA PFDD Meeting

MDA Is proud to have collaborated with Cure SMA and SMA stakeholders in the April 18, 2017 PFDD meeting for SMA.  Twenty different panelists, representing both patients and caregivers with various types, ages and stages of SMA, testified as part of the meeting, which was followed by a period of facilitated discussion and polling questions.  More than 400 individuals participated in the meeting, both in-person and via webcast, all of whom shared information about their experiences to help FDA officials understand the progress of the disease, as well as the impact and needs of those living with SMA.  MDA appreciates the efforts to everyone who participated and to those who helped make the event such a success.  The information captured at the meeting was published in a document called The Voice of the Patient Report and submitted to the FDA to help evaluate future SMA therapies.

CMT PFDD Meeting

MDA partnered with the Hereditary Neuropath Foundation (HNF) and other CMT stakeholders on the September 28, 2018 PFDD meeting for CMT.  The meeting was a productive opportunity for CMT patients, caregivers, and advocates to connect and inform the FDA on the needs of the community.  Hundreds of patients and family members joined the meeting in person and virtually.  MDA was pleased to work with HNF and the Charcot Marie Tooth Association (CMTA) on this meeting and is proud to have contributed to the ensuing Voice of the Patient report. 

Mitochondrial Disease PFDD Meeting

MDA partnered with the United Mitochondrial Disease Foundation and MitoAction to convene the mitochondrial disease community for the “Energy in Action” Patient-Focused Drug Development meeting on March 29th, 2019. Similar to previous PFDD meetings, the viewpoints of the community were captured and published in a Voice of the Patient report on December 3rd, 2019.

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